Showing 390 results for "zolgensma"

MDA Awards $6.6M to Scientists Working in SMA, Other Neuromuscular Diseases

Some $6.6 million in new Muscular Dystrophy Association (MDA) multiyear grants will support promising studies in an array of neuromuscular disorders, including spinal muscular atrophy (SMA). Through their work, the 25 award recipients hope to better understand disease mechanisms, build upon current treatments,  advance the identification of new…

Severity of Motor Problems at Diagnosis Helps to Predict Progression in SMA Type 2, Study Says

The severity of motor problems in children at the time of diagnosis with spinal muscular atrophy (SMA) type 2 may help to predict likely disease progression, including the start of scoliosis and required ventilation, according to an Italian observational study. This is important because a better understanding SMA’s natural history — how…

Russia Approves Spinraza to Treat All SMA Patients

Russia has become the first of the 15 former Soviet republics to approve Biogen’s Spinraza (nusinersen) to treat spinal muscular atrophy (SMA). In its decision, the Russian Health Ministry noted that much more data exists on Spinraza’s benefits for patients 17 years and younger than for those 18…

Bionews’ Patient Forums Foster Community and Raise Awareness

Online forums have markedly altered how patients, caregivers, and healthcare professionals communicate and connect. Even when miles apart, individuals can establish supportive relationships, share experiences and information, and sometimes just vent. Bionews Services, a leading online health, science, and research publication company, has been rolling out its…

The SMA Community Is United in Diversity

I’ve been an active member of the SMA community for years, but one of my fondest memories from any SMA conference took place last year in Dallas, Texas. As I chatted with my friend Doug, another friend’s 5-year-old daughter joined our conversation. Her…